Building global development strategies for cf therapeutics during a transitional cftr modulator era

The cystic fibrosis (CF) community is experiencing transformative changes that will forever alter the landscape of care with the discovery and confirmation of clinically effective CF transmembrane conductance regulator (CFTR) modulator therapies potentially effective for approximately 90% of the CF population [1]. Although tremendously promising, critical goals remain to identify disease modifying therapies for all, develop new therapeutics to address the remaining complications of a multifaceted disease and, equally as important, ensure the equitable access of these therapies to all individuals with CF worldwide.

Source: Journal of Cystic Fibrosis - June 6, 2020 Category: Respiratory Medicine Authors: N. Mayer-Hamblett, S. van Koningsbruggen-Rietschel, D.P. Nichols, D.R. VanDevanter, J.C. Davies, T. Lee, A.G. Durmowicz, F. Ratjen, M.W. Konstan, K. Pearson, S.C. Bell, J.P. Clancy, J.L. Taylor-Cousar, K. De Boeck, S.H. Donaldson, D.G. Downey, P.A. Flume, Source Type: research